
FDA clears two sickle-cell gene therapies, including CRISPR ...
The US FDA announced today that it has cleared two novel gene therapies for sickle cell disease: Vertex Pharmaceuticals' Casgevy and bluebird bio's Lyfgenia.
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The US FDA announced today that it has cleared two novel gene therapies for sickle cell disease: Vertex Pharmaceuticals' Casgevy and bluebird bio's Lyfgenia.
The White House said today that it may take enforced ownership of patents on medicines developed with public funding if their manufacturers set their prices too high.
The FDA has kicked off a priority review of Genfit and Ipsen’s elafibranor as a treatment for rare cholestatic liver disease primary biliary cholangitis (PBC), setting a d
Novartis’ targeted factor B inhibitor iptacopan has become the first oral monotherapy to be approved by the FDA for rare blood disorder paroxysmal nocturnal haemoglobinuri
While global harmonisation remains the holy grail, the reality is that pharmaceutical companies still face significant challenges navigating regulatory differences.
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